CN101926824A - 用于遗传修饰造血祖细胞的方法和这些被修饰细胞的应用 - Google Patents

用于遗传修饰造血祖细胞的方法和这些被修饰细胞的应用 Download PDF

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CN101926824A
CN101926824A CN2010102736257A CN201010273625A CN101926824A CN 101926824 A CN101926824 A CN 101926824A CN 2010102736257 A CN2010102736257 A CN 2010102736257A CN 201010273625 A CN201010273625 A CN 201010273625A CN 101926824 A CN101926824 A CN 101926824A
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hematopoietic cell
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transduction
compositions
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杰弗里·P·西蒙兹
拉斐尔·阿马多
孙伦康
珍妮特·麦克弗森
格雷格·范宁
韦恩·格拉赫
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Johnson and Johnson Research Pty Ltd
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

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  • Tropical Medicine & Parasitology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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CN2010102736257A 2001-07-10 2002-07-10 用于遗传修饰造血祖细胞的方法和这些被修饰细胞的应用 Pending CN101926824A (zh)

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US30412701P 2001-07-10 2001-07-10
US30428301P 2001-07-10 2001-07-10
US60/304,127 2001-07-10
US60/304,283 2001-07-10
US34348401P 2001-12-21 2001-12-21
US60/343,484 2001-12-21
US38606302P 2002-06-04 2002-06-04
US60/386,063 2002-06-04

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CNA028177428A Pending CN1553963A (zh) 2001-07-10 2002-07-10 用于遗传修饰造血祖细胞的方法和这些被修饰细胞的应用

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EP (2) EP1414996B1 (enExample)
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KR (1) KR101015913B1 (enExample)
CN (2) CN101926824A (enExample)
AT (1) ATE490322T1 (enExample)
AU (1) AU2002316640C1 (enExample)
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CA (1) CA2453531A1 (enExample)
DE (1) DE60238486D1 (enExample)
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113897398A (zh) * 2012-09-07 2022-01-07 再生元制药公司 经遗传修饰的非人动物及其使用方法
CN114766432A (zh) * 2012-11-05 2022-07-22 再生元制药公司 经遗传修饰的非人动物及其使用方法

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113897398A (zh) * 2012-09-07 2022-01-07 再生元制药公司 经遗传修饰的非人动物及其使用方法
CN114766432A (zh) * 2012-11-05 2022-07-22 再生元制药公司 经遗传修饰的非人动物及其使用方法

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WO2003006691A1 (en) 2003-01-23
IL159637A (en) 2013-07-31
KR20040032852A (ko) 2004-04-17
JP2010077125A (ja) 2010-04-08
BR0211079A (pt) 2005-08-30
CA2453531A1 (en) 2003-01-23
US7345025B2 (en) 2008-03-18
EP1414996A1 (en) 2004-05-06
EP2322618A1 (en) 2011-05-18
AU2002316640C1 (en) 2009-01-29
JP2005528319A (ja) 2005-09-22
CN1553963A (zh) 2004-12-08
IL159637A0 (en) 2004-06-01
US7776595B2 (en) 2010-08-17
NZ530624A (en) 2009-02-28
MXPA04000268A (es) 2005-03-07
JP5179697B2 (ja) 2013-04-10
US20080044394A1 (en) 2008-02-21
DE60238486D1 (de) 2011-01-13
KR101015913B1 (ko) 2011-02-23
US20040072771A1 (en) 2004-04-15
EP1414996A4 (en) 2007-03-28
AU2002316640B2 (en) 2008-06-12
EP1414996B1 (en) 2010-12-01
HK1065074A1 (en) 2005-02-08
DK1414996T3 (da) 2011-03-21
ATE490322T1 (de) 2010-12-15

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